Polynucleotides encoding methylmalonyl-coa mutase for the treatment of methylmalonic acidemia
OwnerHusain Attarwala, Kimberly Ann Coughlan +3inventors
InventorsHusain Attarwala, Kimberly Ann Coughlan, Ruchi Jain, Matthew Lumley, Vladimir Presnyak
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Abstract
This disclosure relates to mRNA therapy for the treatment of methylmalonic acidemia (MMA). mRNAs for use in the invention, when administered in vivo, encode methylmalonyl-CoA mutase (MUT). mRNA therapies of the disclosure increase and/or restore deficient levels of MUT expression and/or activity in subjects.
Claims50
A method of treating methylmalonic acidemia in a human subject in need thereof, the method comprising administering to the human subject by intravenous infusion a lipid nanoparticle comprising an open reading frame (ORF) encoding the human methylmalonyl-CoA mutase (MUT) polypeptide of SEQ ID NO:1, wherein the ORF is at least 96% identical to the nucleotide sequence of SEQ ID NO:7, and wherein the mRNA is administered at a dose of 0.01 mg/kg to 2.0 mg/kg.
Dependencies are inferred from the claim text.